Selecting informative patients for phase 2 progressive trials in MS: Design considerations for phase 2 clinical trials in progressive MS.

IF 4.8 2区 医学 Q1 CLINICAL NEUROLOGY
Marcus W Koch, Carlos Camara-Lemarroy, Eva Strijbis, Jop Mostert, Victoria M Leavitt, Pavle Repovic, James D Bowen, Jacynthe Comtois, Bernard Uitdehaag, Gary Cutter
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引用次数: 0

Abstract

While relapsing-remitting multiple sclerosis (MS) has many therapeutic options, progressive forms of MS remain largely untreatable. Phase 2 clinical trials are our main tool to advance new treatments for progressive MS. Given the complexities of progressive MS, it will likely require many phase 2 trials to improve its treatment. To conduct informative and efficient phase 2 trials, it is important that such trials are designed in a way that they can identify a successful treatment as quickly and with as few participants as possible. In this topical review, we discuss cohort selection, outcome selection, cohort enrichment, and dosing selection as strategies to optimize the efficiency of phase 2 clinical trials in progressive MS.

为多发性硬化症进展期 2 期临床试验选择信息灵通的患者:进展期多发性硬化症进展期 2 期临床试验的设计注意事项。
虽然复发缓解型多发性硬化症(MS)有很多治疗方案,但进展型多发性硬化症在很大程度上仍无法治疗。2 期临床试验是我们推进进展型多发性硬化症新疗法的主要工具。鉴于进展型多发性硬化症的复杂性,很可能需要多次 2 期临床试验才能改善其治疗效果。要想开展内容翔实、效率高的 2 期临床试验,重要的是此类试验的设计应能尽快确定成功的治疗方法,且参与人数越少越好。在这篇专题综述中,我们将讨论队列选择、结果选择、队列富集和剂量选择等策略,以优化进展期多发性硬化症 2 期临床试验的效率。
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来源期刊
Multiple Sclerosis Journal
Multiple Sclerosis Journal 医学-临床神经学
CiteScore
10.90
自引率
6.90%
发文量
186
审稿时长
3-8 weeks
期刊介绍: Multiple Sclerosis Journal is a peer-reviewed international journal that focuses on all aspects of multiple sclerosis, neuromyelitis optica and other related autoimmune diseases of the central nervous system. The journal for your research in the following areas: * __Biologic basis:__ pathology, myelin biology, pathophysiology of the blood/brain barrier, axo-glial pathobiology, remyelination, virology and microbiome, immunology, proteomics * __Epidemology and genetics:__ genetics epigenetics, epidemiology * __Clinical and Neuroimaging:__ clinical neurology, biomarkers, neuroimaging and clinical outcome measures * __Therapeutics and rehabilitation:__ therapeutics, rehabilitation, psychology, neuroplasticity, neuroprotection, and systematic management Print ISSN: 1352-4585
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